Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.
about
Enzyme replacement and substrate reduction therapy for Gaucher diseaseClinical studies in lysosomal storage diseases: Past, present, and futureOrphan therapies: making best use of postmarket data.Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatmentCost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease.Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation.Quality of life in patients with Fabry disease: a systematic review of the literature.An evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher diseaseA Dysmorphometric Analysis to Investigate Facial Phenotypic Signatures as a Foundation for Non-invasive Monitoring of Lysosomal Storage Disorders.Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain.Seven-year safety and efficacy with velaglucerase alfa for treatment-naïve adult patients with type 1 Gaucher diseaseA population-based registry as a source of health indicators for rare diseases: the ten-year experience of the Veneto Region's rare diseases registry.Natural course of Fabry disease and the effectiveness of enzyme replacement therapy: a systematic review and meta-analysis: effectiveness of ERT in different disease stages.Treatment of lysosomal storage disorders: successes and challenges.Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.Hearing loss in children with Fabry disease.Pediatric registries at the Food and Drug Administration: design aspects that increase their likelihood of success.Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factorsThe history and accomplishments of the ICGG Gaucher registry.Ethical, legal and social implications of rare diseases and orphan drugs in Europe: meeting report of a Brocher symposium.Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.Rare disease registries: potential applications towards impact on development of new drug treatments
P2860
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P2860
Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders.
description
2011 nî lūn-bûn
@nan
2011 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Limitations of drug registries ...... f lysosomal storage disorders.
@ast
Limitations of drug registries ...... f lysosomal storage disorders.
@en
type
label
Limitations of drug registries ...... f lysosomal storage disorders.
@ast
Limitations of drug registries ...... f lysosomal storage disorders.
@en
prefLabel
Limitations of drug registries ...... f lysosomal storage disorders.
@ast
Limitations of drug registries ...... f lysosomal storage disorders.
@en
P2093
P2860
P356
P1476
Limitations of drug registries ...... f lysosomal storage disorders.
@en
P2093
Carla E M Hollak
Jeremy Manuel
Johannes M F G Aerts
P2860
P2888
P356
10.1186/1750-1172-6-16
P5008
P577
2011-04-16T00:00:00Z
P5875
P6179
1003424485